FDA Benefit-Risk Assessment

The National Organization of Rare Disorders (NORD) has distributed the following message to its members.  The KDA participated in the initial solicitation of support, but the current proposed guidelines falls a little short by not asking for patient input.  For those of us with Kennedy's Disease, ALS or MS, for example, I believe we would like a say in this process ... especially as the disease progresses.

Below is NORD’s message plus the link to their letter:

Update on Benefit-Risk Assessments

Benefit-risk assessments are the basis of FDA's regulatory decisions in the pre-market and post-market review process of drugs and biologics. The patient voice in this process is critical. In our letter to the FDA September 27, 2011,we expressed our hope that patients and patient organizations would be able to contribute toward the agency's decision-making in assessing the benefit-risk equation of new products as well as the amount of risk patients at various stages of their condition are willing to take, the quality-of-life challenges they face, the ways they receive information about the proper use of their therapies, how often they see and receive information from their physicians, and other information that FDA medical reviewers and other relevant FDA staff may benefit from knowing directly from patients.

The FDA published a draft of their “Proposed Approach to Benefit-Risk Assessment in Drug Regulatory Decision-Making” in February 2013.  Unfortunately, this draft- as currently written- falls short of the expectations to include the patient voice in the process.

Please review our comments to the FDA, which are due May 7.  Your voice and support of these comments is critical.  To sign on to these comments, please contact Diane Dorman, ddorman@rarediseases.org, no later than Monday, April 29. If you have additional questions, please do not hesitate to contact her.

View the letter.
 
A paragraph from the linked letter needs reinforcing.
 
“We believe that the benefit-risk framework would be substantially improved if it were revised to include the meaningful and timely involvement of patients. FDA should also establish mechanisms to communicate with patients, and receive information from patients, to enhance the factual basis of individual benefit-risk assessments in real time. Such a mechanism would serve FDA’s goal of avoiding incorrect assumptions about patient risk tolerance, as well as providing the contextual information that is key to the benefit-risk framework. We also believe that FDA can, and should, exercise leadership to include patients and to include the regulated industry which has the resources to develop the kinds of contextual data that FDA seeks. As new drugs, particularly those to address unmet medical needs, are continuously being offered for review, FDA needs an enhanced timetable for assuring patient input.”

If interested, I recommend that you contact your Senators and Representatives expressing your support of ‘patient involvement’ in the benefit-risk assessment process.